AB1496 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH HIDS (HYPER-IGD SYNDROME) − INTERIM ANALYSIS OF THE RELIANCE REGISTRY
Authors: P. Oommen, T. Kallinich, J. Rech, N. Blank, J. Weber-Arden, J. B. Kuemmerle-Deschner
Journal: Annals of the Rheumatic Diseases (2023)

<h3>Background</h3> Hyper-IgD Syndrome/Mevalonate Kinase Deficiency (HIDS/MKD) is a rare autoinflammatory disease caused by a defect in the gene encoding mevalonate kinase. Treatment with the interleukin-1β inhibitor canakinumab (CAN, approved since 2017 for HIDS/MKD) resulted in rapid remission in most patients, both in controlled trials and in clinical practice. <h3>Objectives</h3> The present study investigates the long-term efficacy and safety of CAN under routine clinical conditions in pediatric (age ≥2 years) and adult patients with HIDS/MKD. <h3>Methods</h3> RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany. Patients with clinically confirmed HIDS/MKD diagnosis who routinely receive CAN are enrolled in the study. Disease remission, disease activity, fatigue, days absent from school/work due to study indication, and inflammatory markers are assessed at baseline and at 6-monthly intervals. <h3>Results</h3> The present interim analysis includes baseline (N=8) as well as preliminary 18-month (N=6) and 30-month data (N=5) from HIDS/MKD patients enrolled in the study by December 2022. Of these patients, 63% were female and median age at baseline was 8 years (2−39 years). The median duration of prior CAN treatment at study entry was 1.5 years (0−5 years). The majority of patients has been treated with greater than standard dose CAN (5/8 patients at baseline, 4/6 at month 18, 3/3 at month 30). Preliminary results indicate stable remission and disease control as assessed by physicians and patients and according to laboratory parameters (<b>table 1</b>). Even though efficacy outcomes in the HIDS cohort are based on small patient numbers, data resemble outcomes in larger cohorts within the RELIANCE registry (CAPS, TRAPS, FMF). Serious adverse events occurred in N=2 patients, but none were considered as drug related. <h3>Conclusion</h3> The present data from baseline and preliminary interim analysis suggest good disease control in HIDS/MKD patients participating in the RELIANCE study. In addition, data analysis at 18 and 30 months revealed no unexpected safety concerns. Further interim and end-of-study data will be analyzed to assess efficacy and safety of long-term CAN treatment in HIDS/MKD patients in the course of the study. <h3>REFERENCES:</h3> NIL. <h3>Acknowledgements:</h3> NIL. <h3>Disclosure of Interests</h3> Prasad Oommen Grant/research support from: Novartis, Tilmann Kallinich Speakers bureau: Roche, Jürgen Rech Speakers bureau: AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Consultant of: AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD, Mylan, Novartis, Roche, Sanofi, Sobi, UCB, Grant/research support from: Novartis, Sobi, Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi, Julia Weber-Arden Employee of: Novartis, J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi.